CMS launches Cell and Gene Therapy Access Model to expand Medicaid access to gene therapies for sickle cell disease

New model introduces outcomes-based agreements to balance affordability and access for high-cost therapies

The US Centers for Medicare & Medicaid Services (CMS) has launched its Cell and Gene Therapy (CGT) Access Model, a novel initiative designed to improve Medicaid patients’ access to innovative gene therapies for sickle cell disease (SCD) while managing costs for state programs. Originally introduced by the Biden-Harris Administration, the model allows CMS to negotiate outcomes-based agreements (OBAs) directly with manufacturers on behalf of participating state Medicaid agencies.

Led by the Center for Medicare and Medicaid Innovation, also known as the CMS Innovation Center, the model brings together 33 states, the District of Columbia, and Puerto Rico, covering approximately 84% of Medicaid beneficiaries with SCD. By linking pricing to patient outcomes, CMS aims to improve access to potentially curative therapies while supporting state budgets.

“This model is a game changer,” noted CMS Administrator Dr Mehmet Oz. “CMS is giving states the tools to deliver lifesaving therapies to patients in need, while holding manufacturers accountable for outcomes and protecting taxpayer dollars.”

SCD affects ~100,000 Americans, disproportionately impacting Black and Hispanic communities. Medicaid covers ~50–60% of individuals living with SCD, and the disease accounts for nearly $3 billion annually in Medicaid-related healthcare costs, driven by frequent hospitalizations and long-term complications such as stroke, organ damage, and chronic pain.

Until late 2023, the only curative option for SCD was allogenic stem cell transplantation. The FDA’s approval of two gene therapies – CASGEVY™ (Vertex Pharmaceuticals/CRISPR Therapeutics) and LYFGENIA™ (bluebird bio, Inc.) – represented a significant advance in treatment options. Priced at $2.2 million and $3.1 million per patient, respectively, the therapies offer transformative potential but raise significant affordability challenges.

A policy analysis from the Institute for Clinical and Economic Review (ICER) and the NEWDIGS FoCUS Project published in the Journal of Comparative Effectiveness Research highlights three key challenges for gene therapies: determining fair pricing, managing clinical uncertainty, and addressing short-term budget impacts. “Given the potential impact of these therapies on human lives, novel solutions to these challenges must be considered to achieve the right balance of providing incentives for innovation while ensuring equitable and affordable access for health systems and for patients,” the analysis noted.

These considerations underscore the need for new market and policy approaches as more gene therapies are approved, including those for conditions affecting larger patient populations. As Tyler D Wagner and colleagues from the National Pharmaceutical Council noted:

“The unique characteristics of gene therapy treatments, in conjunction with uncertainties about long-term durability and future market competition, necessitate the development of innovative models to assess the value of these treatments, promote appropriate payment and advance meaningful patient access while maintaining incentives for future research and development.”

By negotiating OBAs directly with manufacturers on behalf of states, CMS aims to address longstanding market access challenges associated with the high cost of cell and gene therapies. These OBAs tie payment to patient health outcomes over a defined period. If the therapies do not deliver expected clinical benefits, manufacturers must provide rebates or other price concessions. The initiative could serve as a blueprint for future payment models in the US, where budget impact concerns and uncertainties around long-term effectiveness often hinder payer uptake of innovative treatments.

The model provides:

• CMS-negotiated OBAs developed with input from states, patients, and providers.
• Optional federal funding of up to $9.55 million per state to support implementation, outreach, and data tracking.
• Flexible start dates between January 2025 and January 2026.
• Potential future expansion to include other high-cost therapies beyond SCD.

Participation is voluntary for both states and manufacturers. Bluebird bio and Vertex Pharmaceuticals have already entered into separate agreements with CMS, committing to OBAs under the model.

A notable feature of the CGT Access Model is its focus on collecting real-world data to assess the therapies’ impact over time. CMS is partnering with the Center for International Blood and Marrow Transplant Research (CIBMTR) to gather both clinical outcomes and patient-reported data. Treatment centers administering gene therapies under Medicaid will need to participate in this data collection effort. For manufacturers, this creates both an opportunity and a requirement: to support payer demands for evidence while demonstrating long-term durability and value. The framework also reflects a broader industry trend of integrating real-world evidence into pricing and access strategies.

While the initiative has been praised as a “major win for American patients” and a potential blueprint for future high-cost therapies, questions remain about how it will interact with broader Medicaid reforms. Legislative changes set for 2026 under the “Big Beautiful Bill” could affect eligibility and coverage for millions of Medicaid beneficiaries, potentially complicating access gains from the model.